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tv   Charlie Rose  PBS  January 3, 2015 12:00am-1:01am PST

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>> charlie: welcome to the program. we begin this new year with a look back at one of my favorite broadcasts in 2014. it is about the brain and new add -- advances and understanding in treating blindness. the brain has been a particular focus of this program and so we look back at that program but think about the future and the remarkable achievements in science. >> as you indicated, there are close to 300 million people worldwide that have various degrees of visual impairment and, in the past, the only thing you could do for people like that is to give them non-visual guides. teach them how to read braille, a seeing eye dog. but recently this has changed. we are sitting here amidst the revolution of treatment of mack what are degeneration and it opens the treatment of many kinds of blindness.
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that is because three major developments have occurred -- gene therapy, stem cell therapy and red fill chips. >> charlie: >> rose: funding for "charlie rose" has been provided by: >> rose: additional funding provided by: >> and by bloomberg, a provider of multimedia news and information services worldwide. captioning sponsored by rose communications from our studios in new york city, this is charlie rose. >> charlie: to be blind is nod miserable. not the be able to bare blindness, that is miserable. john milton wrote that more than
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285 million people currently live with visual impairment. for many of these cases, there is no cure, yet in recent years there have been breakthroughs in our understanding and treatment of blindness. sanford greenberg lost his vision to glaucoma at age 19. he is chairman of the board of governors of johns hopkins wilmer eye institute. he talks about his experience and mission to end blind mess. also joining me jean bennett, university of pennsylvania, steven schwartz, jules stein eye institute, eberhart zrenner, university of tubingen and carla shatz, stanford university school of medicine and eric kandel, columbia university. i begin with eric, giving an overview of our subject tonight. eric. >> charlie, the last program we did was new approaches in the treatment of deafness. tonight it's new approaches in
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the treatment of blindness. as in the case of deafness blindness is not a life-threatening situation but it's tremendously disabling and in some ways more disabling than deafness because as you pointed out, there are a number of very important blindness conditions for which there is no treatment. now, why is that so? unlike deafness, the sensory organ of the vision, the rete any, which lines the inside surface of the eye, that is the most complex sense organ that we have. in fact it's not a peripheral organ. it's actually a part of the central nervous system. it's an extension of the central nervous system and as a result it has the complexity of central nervous system structure. it's not uniform. it has a small area in the center that is clear on that image which is called the macular. the macular is the area of
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greatest visual acuities. if i focus in on you i turn my head toward you my eyes focus on you and the macular really analyzes your features. unfortunately, the cells of the macular are extremely sensitive to damage that leads to blindness, and, so, this is a really serious problem and we at the moment, have no treatment for that, even though this is a point of great spatial acuity. blindness, as you indicated is a range of conditions ranging from complete to partial blindness. complete blindness means you don't see images, you don't see figures, you don't see people, you can't tell the difference between day and night. this is sandy's condition. partial blindness varies from tunnel vision to cloudy vision to having night blindness. now, you can have difficulty
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with vision from two sources one is if you have a damage to the visual pathway that goes from the retina into the brain and carla will discuss that more, but we are not going to focus in on that as a source of blindness. we're going to limit ourselves to disorders of the retina. and, you know, as you indicated, there are close to 300 million people worldwide that have various degrees of visual impairment and they fall into two categories -- treatable diseases and, at the moment things that are essentially untreatable. so cataracts, glaucoma and diabetic retinopathy are treatable. can catted racquets, cloudy vision glaucoma tunnel vision and chi becket rete knoply you -- diabetic rete retinopathy, you have problem with night vision.
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these are imminently treatable conditions in cases of diabetic retinopathy, these are treatable diseases in. the undeveloped world, they are major forces of blindness because people don't have access to care. in the developed world, the major problem is macular degeneration. as we indicated, there's a form that accounts for most of it called dry macular degeneration which is really sort of an age-related disorder in which people really lose a lot of their vision, their visual acuity. in the past the only thing you could do for people like that is to give them non-visual guides, teach them how to read braille, a seeing-eye dog handrails in their apartment, and speech compression devices of the kind sandy developed which makes it easy for them to handle auditory information. but recently, this has changed. we are sitting here in the midst
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of a revolution in the treatment of macular degeneration, and it opens the treatment of many kinds of blindness. that is because three major developments have occurred. gene therapy, stem cell therapy and retinal chips. gene therapy is an attempt to replace a defective gene with a normally functioning one. genes are not functioning and one can insert a normal functioning gene into the cell and in many cases help the situation. with stem cell therapy you're rescuing a whole cell type, not just a gene. for the last 15, 18 years it's been possible to take stem cells that can take on the properties of any cells in your body and with the appropriate chemical mixture get them to be retinal cells of various kinds and
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replace the defective cell line. with retinal chips, you implant that deep in the retina and you stimulate directly electrically pathways that lead to the brain stimulating neural elements that lead to the brain. and we have with us by coincidence the three pea near as in --pioneers in this area. jean bennett pio neared the study of gene therapy. steve pioneered t stem cell therapy. eberhart zrenner is one of the outstanding leaders in developing retinal chips, the microchips that are amazing for people who can't respond to the other treatments. we have the privilege of having two other people here. carla shatz is the leader of the study of the visual system and
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my friend sandy here is amazing. he as you pointed out, is a college undergraduate, had a severe case of glaucoma for which he became blind and he will tell us what it's like to be blind. but the amazing thing to me about sandy is, despite the fact he has this tremendous handicap, he's a remarkable human being. he's had a rich happy, productive life. he's invented things and now recently has begun to think of how he can help other people who are blind to make their life better. >> charlie: sandy great to have you at the table. talk to us about becoming blind and the whole sense of the journey for you. >> well, even after 50 years i can still feel what it was when i went blind. for many months i had declining vision.
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my mother and i went to see a glaucoma specialist. he examined me and turns to me with my mother in the room and says well, son, you're going to be blind tomorrow. i guess that was my moment, and i think there is not a moment that occurs in just -- i think there is a moment that occurs in just about everyone's life that the instant before bad news is given, after which nothing else in your life will be the same, and after which you look back on your life and say my god i didn't realize how good i had it until now. before that moment, all for me, anyway was possible and all was rather self-evidently actual. after that moment zero possible, zero actual.
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one moment you're at the top of your game and the next moment there is no game. after surgery, i felt empty eviscerated my vital parts cut out. and there was a fair amount of pain in my eyes but nothing in comparison with the pain in my heart knowing that my mother had just witnessed her 20-year-old son go blind, you know, his eyes cut open. so, you know, there was really no reason to live. so i prayed, you know in my own way. my girlfriend sue i was convinced, was going to leave me. after all, i was a dropout, had no money no eyes, no future.
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but she didn't and it is really because of her that i'm here tonight with you. she and my college roommate art garfunkel set me really on the way out of my horrific wilderness. i returned to columbia. then something happened then not too far from where we're sitting tonight that turned me away from blindness. art and i were walking toward grand central station during rush hour when he abandoned me. so i got down to the steps to that hole in the ground on my own, grand central station at rush hour, and you're blind. so i stumbled to the train that
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got me back to columbia. and as i walked through the large iron gates of that great university a guy bumps into me and says, oops, excuse me, sir. it was, of course, you know, my roommate this guy called garfunkel. and he had not abandoned me. he followed me the entire way. as i bumped into his chest that instant, i knew that if i could get through the new york city subway system blind, there were no limitations to what i could accomplish. >> that's great. all was possible for me. >> charlie: so what do you hope for today?
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>> what do i hope for today is that eric and the people who are sitting at this table who, in my view embody the achievements of human genius, for them to end this plague. and it's been 6 million years since we humans and our ancestors have been afflicted by this thing. it's got to end, and a group of us have started something called end blindness by 2020, and, you know, sue and my family and my college roommates, art and jerry spire, he was actually a witness to my subway odyssey, who have been with me and for me since we both entered columbia college together, we launched an effort a few years ago to end blindness
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by 2020. >> you know, sandy what i find very interesting about you -- first of all, i must say having art and jerry spire as roommates is a very good beginning to life from any point of view. (laughter) but how did you accomplish what you accomplished with this handicap? i mean, you went to harvard, you made a major invention, you've led a rich life, you're happy married. >> well, i told you, the centerpiece is sue, and then my family, my children, paul, jimmy and katherine have stuck by me and these two guys who as you say, i was really fortunate enough to meet when we all started columbia together. but, you know it's not all black or dark. i don't want to leave that impression with you because when
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you're not distracted by visual images, you develop a life within your mind. so i can see you now, eric, the way i saw art back before i was 19 here in this city and you look pretty damn good you know. (laughter) >> charlie: your mind is playing tricks on you. >> charlie, you look like the david from the shoulders up. (laughter) >> charlie: sandy, that's remarkable. you are why we do this television program, so thank you. let me turn to carla. give me an overview so we understand exactly what it is we're talking about in terms of the human eye. >> well vision is really a miraculous process and to hear
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about losing it is also very heart-rending. thank you so much, sandy for what you said. and i want to tell you a little bit about the visual system and talk about the basic layout of the system before we get into really talking about the eye. of course vision starts in the eye, and light enters the eye, and there's a special layer at the back of the eye which is called the retina. but you can think of the retina as kind of a fancy digital camera. and the light-sensitive part of the eye, you can kind of think of it as like the pixels in your camera though the pickles are made out of silicone but in your eye the pixels are the nerve cells and very specialized nerve cells which absorb light and convert it to a neural signal. and then the information is sent from the retina -- as you can see the arrows -- to structures in the central part of the
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brain. this is called the central visual pathways. the first place is the lateral geniculate nucleus and the second is the pvimary visual cortex. we talk about the visual camera idea. s in kind of like the central processing unit where a lot of image analysis happens so it starts in the eye, but most of the information processing and data crunching for the visual system is happening in the central visual pathways. now, you can ask what is the nature of the information that comes out of the eye? what's it like? and i was thinking about how to convey this, and i thought i can't do it any better than george. so there's this wonderful sera painting. you will notice in the painting the world is broken up into
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thousands of dots of color. if you stand up really close you put your nose to the painting basically you're just going to see a lot of dots, but the remarkable thing is if you stand back away from the painting what you actually see is a seated woman. and if we talk about, again this idea of the retina versus the central visual pathways, the concept here is that the retina is sending thousands of dots to the central visual pathways, and, in fact, each dot is carried by a single nerve fiber, and there are about a million of these nerve fibers going to the central visual pathways. so it's the job of the retina actually to deconstruct the visual world into a pixilated view of the world. so thousands of pixels. and it's the job of the central visual pathways to reconstruct the world by making a kind of a
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seamless view of the world again, kind of connecting the dots back. so we really need our central visual pathways to appreciate that this is a woman seated. but we only need our retina to appreciate the dots. so this is kind of that seeing starts in the eye but really it's the computing power of the rest of the visual system that's needed for what you could call visual perceptions. you can see but you need the central pathways for visual perception. damage to any part of this pathway will produce blindness. so damage to the eye will produce blindness and also damage to any of these other connections will produce blindness. but what we really want to talk about today is blinding eye diseases. so i would like to get more nitty gritty and talk about what's in the eye. if you look on the left, you see that light comes through the
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lens, and just like a camera the lens is focusing the light on the back of the eye, on the structure called the retina. you will see that the back of the eye, the retina, is not uniform. you will see that there is kind of a dip or a pit that's present in the eye. this is called the macular region of the retina, and it's in this region that we have our highest resolution vision. by the way it's also part of the retina that lets you see a color. the other part of the retina outside of the macula is called the peripheral retina. and this is very important, too, because it's the part of the retina that lets us see in the dark. so this is low-light vision. actually, you can appreciate this part of the retina too, because if you've ever looked at a starry night, if you notice if you don't point your eye
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directly at the star but look a little off kilter, the star gets brighter and that's because you'reym])nĂ· the peripheral retina, the edges of the retina which has very high sensitivity for light but isn't as good for high resolution images. so we have these two aspects of the retina. now, the last thing i want to talk about is what are the types of neurons that are in the retina. and so, we can look at a blowup of this macular region. there are nerve cells specialized nerve cells in the retina capable of converting light energy to a neural signalnc and those are called photoreceptors. there are two kinds of photoreceptors. there are the rods and the cones. the cones are the photoreceptors that are crucial for high-resolution vision and they're in the center, the macula. the rods are the photoreceptors that are in the peripheral retina, and those are really
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essential for high sensitivity vision. the next step of visual information processing is that the information from the photoreceptors is passed along to the bipolar cells and from the bipolar cells the information is passed along to the ganglion cells, and the ganglion cells are the output neuron of the eye. so they're the nerve cells that send a million long fibers out into the optic nerve and into the optic track into the central visual pathways. one last very important point i want to make and that is there's another critical cell type in the retina. it's called the pigment epithelium and the pigment epithelial cells essentially provide crucial support and nutrients to the photoreceptors, and they're really essential for photoreceptor health and photoreceptor survival.
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and several blinding eye diseases involve and their treatments involve these pigment epithelial cells in the retina. we're going to hear about these cells. i think they're going to feature really quite large in our conversation. >> charlie: that's enormously helpful. thank you very much, a huge understanding of how we see and how important the brain is and how important the pathways are. i want to come now to jean and talk about -- we mentioned gene therapy, stem cell therapy and other things. how can gene therapy be helpful? >> well, carla set the stage for this perfectly because she explained how the pigment epithelium provides a nerve function for the photoreceptors, provides nutrients to the cells, taking away waste products, and the two cell types are interdependent so that if there's a problem in function of one particular gene and one of those cells it affects the other cells secondarily. so in the next image is an
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illustration of one particular example where a mutation can cause disease in both of these cells. you see some pigment epithelial cells next to the photoreceptor cells which are stunted because they're sickly, and the pigment epithelium cells themselves are sickly, they're accumulating liquid droplets because that i have a mutation which prevents them from forming a particular form of vitamin a derivative, which they normally supply to photoreceptors, and that's essential for vision. without the photoreceptors receiving the vitamin a derivative, they can't respond to light and there is no vision, and this makes them sick and they die off as the person ages. and the same thing can go on if there's a mutation in a photoreceptor it can cause disease in the retinal pigment
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epithelium. but knowing this, it's possible -- and knowing the genes which cause these diseases, it's possible to intervene. so i'd like to give you an example of a disease called labors congenital amarosis, which is one of the most severe forms of retinal degeneration, one of the most severe forms of rete us in pig men toe is a mainly does a it's manifest in babies, present at birth. it's usually parents who notice the children aren't responding to visual cues, tools held up to them or shiny objects or miles. instead, the children have abnormal roving eye movements because the retina is not giving signal to the brain and the brain isn't giving signal to the eye muscles to tell them to hold still. this is a disease which has been studied very carefully over the past two decades or so and we now know there are about 19 different genes which can give rise to the same phenotype.
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we've studied one which is called the r.p.e.65 gene. this is one of the more common forms of this condition and there also happens to be an animal model of this disease, puppies born with a spontaneous mutation and they are born blind, and we actually begin our work with these puppies restoring viption to them and reasoned wouldn't it be great to do this with children. so how would you carry out gene therapy? dna the very highly charged. that's a problem for getting a normal copy of dna, a corrective copy across the cell membranes. so you have to be able to encapsulate the normal copy of the gene inside of what's called a recominant virus. we've taken essentially a shell
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of the virus and packaged it with the normal copy of the dna. that dna inside the virus is then injected into the subretinal space which is the space between the neural retina and the nerve cells exposing those cells to the experimental reagent. and in the next image, you can see a closeup of the virus entering the subretinal space and delivering -- entering the retinal pigment epithelium cells. the dna inside that virus then goes into the nucleus of these cells where it sets up shop and starts encoding the protein that is missing which happens to be rp65, in this instance. this is a very stable effect in the first animals that we studied after one single injection. the protein was produced and the dogs could see, after eleven years, and, so, this is really quite stable. so the next question is how
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would this work in children. so the bottom line is that we run a clinical trial using gene therapy for this condition and have found that all of the children involved in this study can now lead essentially normal lives, whereas when they walk into the hospital using a cane or holding their parents' hands because they couldn't see well enough to navigate, they can walk independently, they can sit in the classroom and read books and sit in front of the classroom and see what the teacher is reading on the board, play sports, et cetera, so i would like to show you an example as one of the children. >> you're the pioneer of this. well, my team is. you'll see the video image of an 8-year-old boy who was one of the first children in the world enrolled in a gene therapy clinical trial for a non-lethal
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disease and he is shown three months after he received a single injection in one of his eyes, it happened to be his left eye, and in this video, his injected eye is patched. and what you will see, when this video plays, is that the boy is put through an obstacle course which is in the hospital exam room. it's full of arrows and obstacles that he's supposed to avoid and navigate his way around the course and find a door. what you can see in this video is the boy takes a step and he doesn't know what the do because he can't see anything. and he talks through this video and -- see if you can hear what he says. this is hard. he has to be coaxed. he takes a step and he bumps
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into the object, the sign in front of his eyes because he can't see it. he goes off-course immediately and it takes him a total of 17 minutes to make it through this course. >> charlie: he has great heart, though. >> oh absolutely. on the other hand when his uninjected eye is patched and he's using his newly injected eye, this is the same child, he's walking through the course stepping over an object jutting in his path, avoiding all the obstacles can confident and makes it to the end of the course without any problem whatsoever. so how does this translate into his daily life? this is what is so fantastic. this child who came in using a cane couldn't walk around, is now riding his bike to his friends' houses, playing baseball -- he was on a championship little league team -- hammering objects, playing video games. >> amazing. hitting targets with rocks, maybe doing things his parents would rather him not do but
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essentially leading the life of a normal child. >> charlie: the question we all want to know is to whom is that treatment going to help? what kind of blindness? >> this treatment will be effective for individuals who have mutations in the rpe-65 gene, because that is the type of dna that is delivered into the cells. if they had a mutation in a different gene, that wouldn't be effective. however, the exciting thing is that the same sort of approach can be used to intervene with other diseases that are due to other mutations, and there are now at least a dozen other targets out there, several of which are now in human clinical trials with very exciting results. >> charlie: and they're all learning from each other. >> right. this is is not restricted to the eye. gene therapy and as we'll learn stem cell therapy is used in
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other areas of medicine as well a major advance. >> charlie: talk about stem cell. >> gene therapy is in late-stage clinical trial development and showing remarkable safety, efficacy and durability and meaning it's working well within the clinical trials guidelines and poised, in my opinion, for approval. stem cell or regenerative medicine is different. these studies are early hold great hope and promise but but are in the early stages of development. i would categorize them as highly experimental. appropriately the first stage of any new trial is a safety trial or a phase one trial and that's what we'll talk about today. our hypothesis centers on the idea that we could replace the cells. we could take a stem cell which means that cell is capable of becoming any type of cell in the body. so we could take a stem cell, coax it into becoming a retinal pigment epithelial cell, differentiate, so to speak, and
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take the differentiated cells and inject them into patients who are missing the retinal pigment epithelium with the hope of rescuing or restoring vision. in this our aim is to safely transplant the stem cell retinal epithelial cells. >> she's treating a gene. he's replacing the whole population. >> we're trying to replace it. so for example in macular degeneration, the retinal pigment epithelial cells are lost and we're trying to replace them. we're using a strategy the same as jean. we're taking stem cells and coaxing them into becoming the rpe cells and we harvest them where they're most likely to be
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able to be transplanted into the same space that jean is using in her gene therapy trials to replace the cells. and once they're replaced, they either rescue or restore vision by taking care of the photoreceptors adjacent. >> charlie: i don't understand how you coax them. >> the truth is it's a very surprisingly astonishingly straight-forward method by which stem cells can be induced into certain cell types. it turns out retinal pigment epithelium are relatively low hanging fruit in terms of easy to induce the transformation the a terminally differentiated cell type. it's also important to realize the retinal pigment epithelium are attractive starting for stem cell therapy. kala told us how integrated the retina is. the epithelium has no synaptic connections. it's inside the blood-blaine barrier and protected.
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and it's surgically accessible. so we can get to it surgically relatively safety doesn't require synaptic connections. in the lab we can take the stem cells and turn them into essentially brand new young juvenile retinal pigment epithelium and give someone a fresh layer of cells if they have macular determination say, 40 50, 70 years old, for the duration. we have to date transferred a number of patients. the early studies seem safe seem astonishingly to be giving us a signal there might be some restoration of sings and i wanted to share with you the first person we trance transplant-d young lady who was a set designer and lost her vocation because of her blindness. >> when i was younger, i played a lot of competitive tennis, and i think when i was like in my later teens, i started playing a little more poorly. it was between the lines or the
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ball not quite as, you know, precisely and calling the lines and stuff was a little more difficult. i woke up one morning and i looked across my room and i have a piece of furniture there that's a large armoire and has a lot of carved detail. i had my head to the side and i opened the operated eye and i looked at it. and for the first time i could see the detail in it, but i hadn't been able to see from the distance i was lying down. after that i just got up and i started looking at everything around the house looking at the grass, looking at everything with one eye and then the other eye because they only operated on the one eye. and i could see it a lot better than i had before, and i thought, wow, maybe there's something here that would really be working. it was pretty exciting. >> charlie: how long will it be before you operate on the other eye? >> oh, a long time. we're very very careful
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step-wise, guided by, you know the fda appropriately and by our own ethics committees at u.c.l.a. and other universities. so only the worse eye when we're not certain about safety, as opposed to gene therapy where they're operating on both eyes in these kids. we have not, we're far from that, but we've certainly given this to -- this opportunity to a number of patients. >> how many patients have you done so far? >> between 20 and 30 and they're actually the heros. they're the people who willingly go into this with huge risks and they do it -- >> charlie: what are the risks. >> well, the risks in stem cell and regenerative therapy are enormous. we're careful about making certain we've differentiated the cells into retinal epithelium but if we give them a straight stem cell it could turn into anything the body could reject. so we've had a group of heroic patients to put themselves in harm's way for those who follow.
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>> the encouraging thing is that so far things seem to be moving safely. >> charlie: there are people who don't qualify for either gene or stem cell perp and you've stepped forward for the use of technology. >> right. gene therapy doesn't work, we cannot yet produce photoreceptors, but what we can do is to replace them technically. so we built a chip with photodiodes, and each one takes a spot and amplifies it and sends and electric signal depending on the strength of light. so it's a replacement of the natural photoreceptor with a
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technical photoreceptor. how does it work in we can see this 3 times 3-millimeter chip a little like the chip in the iphone but has a total different life. it has 1,500 photodiodes. >> charlie: 1500. 1,500. the image falls through the lens on to the chip and point by point the image is analyzed turned into an electric mirror image in the bipolar cells, they are stimulated point by point it processes the image and sends the image through the natural pathway of the optic nerve to the output of the retina, back to the central visual system where it is processed in a normal way. but now we have amplifiers there. >> charlie: yeah. they need current. how to do that. so what we see on the left is, again, the retina and the eye, and we put the chip right
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beneath the macular region, that's where we see the best and the brain has learned to see the things. and there is a tiny, yellowish vial going under the retina to the top of the eyeball, crossing the eye, getting out to to empower which goes back to a place behind the ear. but the first loop through on the right side to the retina. on the right is a roundish moon-like optic nerve. left of it is this grey area, a new window of the world to this patient, approximately the size of an ipad in arm length, window size so the patient looks into the world, and the yellowish cable are the power
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supply and the lines to control the chip. you see the head of the patient on the left is an x-ray so there is a comrade chip in the eye and almost a pretzel-like cable that goes to a subdermal receiver you see that behind the ear, in the x-ray and it has a magnet. on the right side if the patient wants to switch on the chip, he simply clips that antenna coil behind the ear the size of a $1 coin and in his hand he has a power box where he can control brightness and contrast like it, an old black and white tv where you have as a contrast and this tiny cable how we ares the antenna and now this reminds us a little bit about the cochlear implant.
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this is much more complex because in hearing we have a signal in time to analyze and here we have to have 1,500 power points to address at the same time. so it's much more complex and more difficult. >> it's a very interesting points. when you speak, there's a sequence to the sounds, and that's much easier to analyze. if you look at a sira painting in which you have to simultaneously analyze all the components of the image, it's much more difficult. >> let's look at the sira painting. how many dots are there? i don't know several thousand. and our chip has, of course, not the power to sort each of those dots so it's probably 20 dots or so which fall on one pixel of our subretinal implant, so the image is not colorful anymore
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because it all mixes up to grey and the borders are not so sharp, as you can say they are a little bit blurred and that's what the patients tell us. and there is a little movie clip next. >> there is the breaks going across there. it's all light up there. and it's lighter there. it's going across. that's the building there. and that's a building that goes up there. but it's not white brick. >> charlie: here she is. she's completely blind. >> completely blind, and she got the chip into the eye, and -- >> charlie: she's seeing what she described the bridge at ox oxford. >> that's what she's seeing through the chip. >> she's describing it.
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in a moment you will see in the next image exactly what that looks like. >> the next imshows what she is describing. it's really an image con consisting of black and white and greyish levels and it's probably not much for us who are seeing so well but for someone who had been blind it makes a lot of a difference to see again at least the surroundings in a blurry, greyish, black and white way and to be able to be confident in walking and we have patients who many are able to see a glass to lift a glass, to see a cup, a knife, a spoon or to go out there in nature and see a goose or something like that. >> charlie: who's eligible for this? >> all the patients who have lost the photoreceptors of the retina, and the retina is still in tact.
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so patients with hereditary retinal degeneration who have lost the rods and the cones and there are one in every 4,000 in new york has this condition. >> charlie: sandy as you're listening to this, tell me what you're thinking. >> well, it's inspiring, and i'm not a guy who likes the "i" word very much, but, you know, there are millions of these children how to there who, you know, where they they are blind and millions will be born who are blind. to make a long story short the people i mentioned before we established a prize to end blindness for the work that best achieves our objective of developing treatment for eye disease, and we just reached
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$3 million in gold bullion as the prize. so we're doing whatever we can to encourage the scientists around the world, and these are without a doubt the leaders. >> what is wonderful about this approach -- >> charlie: which approach? this approach the three of them are taking is that, first of all, it shows there is no single approach that will cure blindness because blindness takes many different forms, and it gets progressively more complicated. jean essentially inserts a single gene into a particular cell type and, boom, he uses stem cells to replace the whole cell population. he takes cases in which you don't even have the epithelial cells. you've lost a good part of the retina and he stimulates the central connections that lead to the central nervous system. so these are progressively more severe diseases that each of these therapeutic approaches
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address. >> charlie: you know as we were watching this, i was thinking of a society that worships athletes and celebrities and sports figures, and here we are with scientists and people who are experimenting with their own lives. >> yes. >> charlie: who give new definition to what it is to be heroic and make real contributions to society. so we're all in your debt. let me just sort of, as we run down the clock here sort of go through each of you and ask about what you hope is possible from the research that you were doing. >> well, on an immediate level, we are running a phase 3 clinical trial, a trial aimed at getting this material approved as a drug so that people who have this condition could benefit from the intervention. this is being run at the children's hospital philadelphia, andeth the only phase 3 gene therapy clinical
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trial going on in the world. and there is no approved gene therapy in the united states and only one other gene therapy approved in the world. we think this could be an approved gene therapy the first approved gene therapy for a blinding condition and could pave the way for opening up the possibilities of developing similar approaches for many other blinding conditions. so we think it's just the beginning. >> i think jean's right. i think gene therapy is here to stay. it's a little like ant bodies were -- antibodies were 20 years ago. it will pave the way for prevalent diseases, not just rare diseases. as far as stem cell i want to continue to emphasize how early it is because i am mindful of not increasing patients' suffering. there is so much stigma about stem cells, people think of a stem cell in the ruth and everyone is cured and the
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fountain of youth, but there is political and social complexities to studying stem cells. we have two political leaders who have contributed to this enormously and from different political parties in. california arnold schwarzenegger started the -- created the california institute for degenerative medicine which paved the way for a lot of this works in california and has translational scientists to stand on the shoulders of great basic scientists in great universities like u.c.l.a. the other is president obama. barack obama is very courageous in leading us following the science and supporting the national institute of health through very complex political and economic times. so to me those could be really important. and i think as jean and eberhart pointed out, it's really the team. i couldn't not do this myself. my hope for the future is work that you do allows people to
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understand the suffering, what sandy has gone through and the countless number of people who go blind unnecessarily bringing blindness to the forefront and ending it is my goal as a treating physician. macular degeneration, whether gene therapy or stem cell therapy or a chip would be a great thing to knock off the podium as sort of the leader of blindness. >> we have operated so far on about 40 patients, and it doesn't work in all of them. some have very degenerated retina and we have to learn to understand why approximately only half to have the patients have useful vision in daily life. but there are more than 30 groups working worldwide on those approaches some of them are in the united states on the cell side others on the brain. especially for the patients like sandy who have lost the optic nerve or other people who have
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lost the eye, it is possible already, at least in my case, to directly connect implants to the brain and that may be a way to help the other patients reach out where it's not a retina situation. of course, there are technical improvements. we cannot only put a single chip in the eye but many chips to increase the visual field and also can transform these complicated pictures by computer software to simple graphs like sketches or caricatures which can be easily grasped. i think there is a lot of room for improvement. >> charlie: car ray, you helped us understand how the brain and retina and eye are connected. as you've sat here today, what would you say is your own hope the future holds? >> i think what we've heard
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today is from three pioneers who have used three different approaches to trying to cure and curing blinding eye diseases, and what's amazing is that the eye is an extension of the brain. it's part of our brain. so what they've done, also, i think, gives us a great deal of hope for dealing with other neurodegenerative brain disorders where nerve cells are lost and where they could be replaced or circuits could even be rebuilt. so my hope is that both from a combination of their work and the work of many other scientists and physicians who are taking -- also working on these problems that soon we will have a lot of options for dealing with the loss of neurons and the brain wherever it happens.
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>> charlie: that's the hopeful note here beyond specifically blindness that the groundbreaking work here can affect a whole range of diseases connected to the brain. >> right. i was thinking, you know, we first discussed deafness, and now blindness, and in both those areas, if you go back 20 years, there was no treatment for almost anything. there was help for deafness with hearing aids that were very primitive. the progress in these areas has been spectacular. what we're talking about here is four or five years old. this is very recent. if you look at my field, psychiatry, you cannot think of the 20 years that have passed as being anywhere comparable. so we really can take hope from these areas in which we've progressed brilliantly that will be app panel to other areas as well. >> charlie: thank you very much. thank you very much.
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thank you for joining us. see you next time. for more about this and earlier episodes, visit us online at and captioning sponsored by rose communications captioned by media access group at wgbh
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this is "nightly business report" with tyler mathisen and soucie. >> stocks out of the gate. first day of trading, 2015. didn't last. by the closing bell s&p 500 did something it hasn't done since 2008. direct response president obama authorizes additional sanctions against north korea in retaliation for the cyber attack against sony pictures. and in the spotlight, five ceos who have their work cut out for them in 2015. all that and more tonight on "nightly business report" for this friday january the 2nd. and i wish you a good evening. happy new year. i'm bill griffeth in